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Researchers engineer in vivo delivery system for prime editing, partially restoring vision in mice

Broad Institute

A team of researchers at the Broad Institute of MIT and Harvard has engineered virus-like particles to deliver prime editors to cells in mice at a high enough efficiency to rescue a genetic disorder. Prime editing, described in 2019 by Liu’s group, can make longer and more diverse types of DNA changes than other types of editing.

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Prime editing efficiently corrects cystic fibrosis mutation in human lung cells

Broad Institute

The new method, published today in Nature Biomedical Engineering , precisely and durably corrects the mutation in human lung cells, restoring cell function to levels similar to that of Trikafta. Next, researchers will need to develop ways to package and deliver the prime editing machinery to the airways in mice and ultimately humans.

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A therapy candidate for fatal prion diseases turns off disease-causing gene

Broad Institute

Unlike gene editing, this “epigenetic” editing does not modify the underlying DNA sequence, but it should switch the gene off permanently, which means that this could be a one-time treatment. They used an engineered adeno-associated virus (AAV) that crosses the blood-brain barrier after intravenous administration. Online June 27, 2024.

Disease 142
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Epigenetic editing: the next generation of genetic medicine

Drug Target Review

The reasons for this are multifaceted, including concerns over the safety of directly altering DNA sequences and subsequent regulatory restrictions that have arisen as a result. The epigenome (meaning ‘above the genome’) is a system of reversible marks regulating how the DNA is read, translated and used. What is epigenetic editing?

DNA 105
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Scientists map the locations of hundreds to thousands of cell types across a mammalian brain

Broad Institute

The other study was led by senior authors Xiao Wang , a Broad core institute member, a Merkin Institute Fellow, and an assistant professor of chemistry at MIT, and Jia Liu, an assistant professor of engineering at Harvard University. The team next applied an approach developed in the Chen and Macosko labs known as Slide-seq.

DNA 140
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Unveiling neoantigen-directed cancer treatment

Drug Target Review

As part of the TCR discovery process and preclinical data package, Adaptive performs rigorous functional characterisation and safety assessments of potent, therapeutic grade antigen-specific TCRs. Genentech is responsible for engineering adaptive-discovered TCRs into T cells and manufacturing the T cell therapy product candidate.

Treatment 105
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Scaling Phage Therapy

Codon

Some algorithms simply look for important similarities between phage and bacterial DNA sequences, a signal of the extensive gene-swapping that goes on when a particular virus and bacteria have shared history. Middle and Right) Once a bacteriophage lands on a cell, it injects its DNA through a tube (purple) into the unwilling host.

Therapies 117